[HTML][HTML] Deciphering conundrums of adeno-associated virus liver-directed gene therapy: focus on hemophilia

GF Pierce, S Fong, BR Long, R Kaczmarek - Journal of Thrombosis and …, 2024 - Elsevier
Adeno-associated virus gene therapy has been the subject of intensive investigation for
monogenic disease gene addition therapy for more than 25 years, yet few therapies have …